Information about "new" treatments for children with autism are brought to the attention of parents through a number of channels such as the news media, word of mouth through other parents, advocacy groups and professional web sites. The amount of information available can be daunting. How are parents to evaluate whether treatments they hear about are appropriate to try for their children?
It is easy to understand that many parents will try an unproven treatment out of desperation based on the small possibility that it will help. But that possible benefit needs to be weighed against the short- and long-term safety of the unproven therapy, as well as cost in terms of a family's time and financial resources. Autism and related conditions include a number of different signs and symptoms, and what is most problematic may differ from one child to the next; it is unlikely that one treatment will be effective for all. Many treatments, such as those related to immunotherapy, eradication of yeast infections, homeopathic herbal treatments, "alternative" medicines, etc., have been proposed, yet none has been studied with the scientific rigor that is necessary to safeguard children or to be certain of real benefit.
It is expected that novel therapies for the treatment of children with autism will be developed at an increasing rate over the next decade. Before a new treatment can be recommended for use in patients, however, it must be shown to be both safe and effective. It is important for parents to understand how new treatments are tested for safety and effectiveness. Scientifically designed clinical trials are the most important--and accepted--means of determining whether a new treatment is safe, whether it works, for what symptoms and in which children.
A clinical trial is a research study designed to answer specific questions about the safety and effectiveness of new therapies as well as to study new ways of using known treatments. Clinical trials are designed to: compare one treatment to another; compare different doses or methods of administering a treatment; or compare a new treatment to no treatment or to customary standards of care. Clinical trials may be used to study drugs as well as behavioral interventions.
Phases of Clinical Trials
Clinical trials are conducted in phases. Each phase is designed to answer different types of questions. In phase I trials, researchers test a new drug or treatment in a small group of individuals, usually healthy volunteers, who do not have the disorder that might be affected by the drug or treatment. The purpose of this phase is to evaluate the drug or treatment's safety, determine a safe dosage range and identify common side effects. In Phase II trials, the study drug or treatment is given to a larger group of people who may have the targeted disorder to see if the drug or treatment has the potential for favorably affecting a specific disorder or condition. Phase II trials also can help in further evaluating the safety and best dose. In phase I and phase II trials, only very common side effects can be identified because the number of subjects is small. In Phase III trials, the study drug or treatment is given to larger groups of affected people to determine its efficacy, identify side effects and collect information that will allow the drug or treatment to be used safely. In some trials, the effects of the drug or treatment are compared to commonly used treatments. Phase IV trials, which are post-marketing studies conducted after the new drug or treatment has been approved for use in patients, provide additional information on the risks, benefits and optimal use of the treatment.
A protocol is a study plan carefully designed to safeguard the participants in the study as well as to answer specific research questions. The study protocol describes: who may participate in the trial; the schedule of tests, visits, procedures, medications and dosages; what outcomes will be measured and how and when they will be assessed; and the length of follow-up. …